National Center for Advancing Translational Sciences (NCATS)

Mission

NCATS' mission is to catalyze the generation of innovative methods and technologies that will enhance the development, testing and implementation of diagnostics and therapeutics across a wide range of human diseases and conditions.

NCATS is all about getting more treatments to more patients more quickly by developing new technologies and operational models to accelerate translation; demonstrating their usefulness in specific applications; and disseminating the approaches, data and methodologies to the broader scientific community.

Rather than targeting a particular disease or fundamental science, NCATS focuses on what is common across diseases and the translational process. The Center collaborates with other government agencies, including other NIH ICs; industry; academia; and patient advocacy groups.

Important Events in NCATS History

December 2011 — NCATS was established on Dec. 23 as part of the Consolidated Appropriations Act, 2012 (P.L. 112-74), which amended the Public Health Service Act.

May 2012 — NCATS and Eli Lilly and Company jointly released an online Assay Guidance Manual. The manual provides researchers with step-by-step guidance through the complex process of turning a basic research finding into an assay that will start the process of discovering pharmacological tools and drugs.

July 2012 — A research collaboration including scientists from NCATS and the University of Wisconsin–Madison helped identify three promising molecular compounds from a collection of approved drugs to pursue as potential treatments for Charcot-Marie-Tooth disease, a genetic neurological disease for which no treatments currently exist.

July 2012 — NIH awarded 17 grants for projects designed to create 3-D chips with living cells and tissues that accurately model the structure and function of human organs, such as the lung, liver and heart. These awards are funded and administered by NCATS. In September 2012, NIH awarded two additional tissue chip grants, administered by NCATS but funded by other NIH ICs.

August 2012 — A team that includes nine NCATS researchers identified compounds that delay tumor formation in mice. The compounds target a specific form of pyruvate kinase, called PKM2, that governs how cancer cells use glucose.

August 2012 — A collaborative research team, including nine experts from NCATS, was honored for its work on an investigational treatment for Niemann-Pick disease type C1, a rare genetic disease of cholesterol storage that eventually leads to neurodegeneration. View Image.

August 2012 — NCATS announced the members of its inaugural Advisory Council and Cures Acceleration Network Review Board.

September 2012 — NIH researchers, including those from NCATS, launched a clinical trial to evaluate the drug candidate DEX-M74 as a treatment for a rare degenerative muscle disease, hereditary inclusion body myopathy (HIBM).

October 2012 — Researchers from NCATS designed a novel drug discovery method that uses two co-expressed reporter genes rather than one to increase the odds of identifying candidate compounds with true activity against biological or disease targets.

November 2012 — NCATS reported that researchers from 13 universities and hospitals, including 10 CTSA Program institutions, partnered with the Cystic Fibrosis Foundation and the drug manufacturer Vertex Pharmaceuticals to conduct clinical trials and obtain Food and Drug Administration (FDA) approval for the drug Kalydeco as a new treatment. View Image.

December 2012 — The NIH Bridging Interventional Development Gaps (BrIDGs) program, administered by NCATS, announced new projects to develop potential treatments for cancers, spinal cord injury and a rare disease.

February 2013 — A team of scientists from NCATS’ Division of Pre-Clinical Innovation and the Laboratory of Viral Diseases at the National Institute of Allergy and Infectious Diseases developed a drug that blocks early-stage herpes simplex virus infections in cultured cells and prevents reactivation of latent virus in mice. The study was published in the Jan. 9, 2013, issue of Science Translational Medicine.

March 2013 — To investigate new ways to treat glaucoma, NCATS partnered with the Johns Hopkins School of Medicine in Baltimore. Led by Don Zack, M.D., Ph.D., a glaucoma specialist and molecular biologist at Hopkins’ Wilmer Eye Institute, the Hopkins team brought extensive knowledge and robust animal models of retinal degenerative diseases. Zack and his team identified several compounds that appeared to stop the death of retinal ganglion cells, the neurons in the back of the eye that, when damaged in glaucoma, lead to vision loss and blindness.

April 2013 — M. Janis Mullaney, M.B.A., officially joined the NCATS team as the associate director for administration.

May 2013 — NCATS published a Federal Register Notice on proposed methods for avoiding duplication, redundancy and competition with industry activities.

August 2013 — NIH announced Extracellular RNA Communications awards designed to improve scientists’ understanding of a newly discovered type of cell-to-cell communication based on extracellular (outside-the-cell) RNA, also called exRNA. View Image.

November 2013 — Scientists at NIH used RNA interference (RNAi) technology to reveal dozens of genes that may represent new therapeutic targets for treating Parkinson’s disease. The findings also may be relevant to several diseases caused by damage to mitochondria. Richard Youle, Ph.D., an investigator at the National Institute of Neurological Disorders and Stroke and a leader of the study, collaborated with NCATS researchers to discover a network of genes that may regulate the disposal of dysfunctional mitochondria, opening the door to new drug targets for Parkinson’s disease and other disorders. View Image.

December 2013 — NIH launched three pre-clinical projects to advance potential new treatments for acute radiation syndrome, brain injury following cardiac arrest and a rare blood disorder called beta thalassemia. The projects are part of NCATS’ BrIDGs program, funded by the NIH Common Fund.

February 2014 — NCATS and the NIH Clinical Center hosted Rare Disease Day to spotlight the challenges encountered by those affected and the significant research and collaboration activities that are helping to make a difference in the development of new diagnostics and treatments.

June 2014 — NIH and NSF collaborated on an I-Corps pilot program to train business-minded biotech researchers. NCATS, the National Cancer Institute, the National Heart, Lung and Blood Institute, and the National Institute of Neurological Disorders and Stroke participated in the program.

July 2014 — The Michael J. Fox Foundation funded a research project that showcases how NCATS' chemical screening resources can advance development of potential therapeutics for a broad range of diseases.

August 2014 — NCATS and National Human Genome Research Institute (NHGRI) scientists developed a potential treatment for patients with Gaucher disease, a rare, inherited condition marked by enlargement of the liver and spleen, anemia, nose bleeds, easy bruising and bleeding, bone problems, and occasionally neurological problems.

September 2014 — NIH funded the next phase of the Tissue Chip for Drug Screening program to integrate tissue chips and test drug effects.

October 2014 — NIH awarded $29 million to expand the Rare Diseases Clinical Research Network to study more than 200 rare diseases.

October 2014 — NCATS announced new research projects to improve treatments for rare blood disorders and infectious diseases. The research, supported through the Center's TRND program, also is designed to provide insights that will broadly improve and accelerate the translational science process.

October 2014 — NIH announced three five-year grants for frontotemporal degeneration research. The projects are being funded by NCATS, the National Institute of Neurological Disorders and Stroke, and the National Institute on Aging.

November 2014 — NCATS experts, academic researchers, a patient advocacy group and a pharmaceutical company collaborated to identify a potential drug target for an inherited neurological disorder called Charcot-Marie-Tooth disease.

January 2015 — NCATS and the Eunice Kennedy Shriver National Institute of Child Health and Human Development entered into an agreement with biotechnology company Vtesse, Inc., to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders. View Image.

January 2015 — NCATS announced the winners of the Toxicology in the 21st Century (Tox21) Data Challenge 2014, a crowdsourcing competition that attracted contestants from 18 countries to design computational models to better predict chemical toxicity.

February 2015 — Extracellular RNA Communication program-supported researchers found that saliva contains many of the same molecules found in blood. The researchers are using these findings to develop a new, noninvasive diagnostic test for stomach cancer. The ExRNA Communications program is supported through the NIH Common Fund.

February 2015 — NCATS and the NIH Clinical Center hosted Rare Disease Day at NIH to raise awareness about these diseases, the challenges that patients face, and the importance of research collaborations.

August 2015 — The White House announced that NIH is expanding its Innovation Corps (I-Corps™) training program to accelerate the commercialization of biomedical technologies developed with federal Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) funding. As part of the expansion, the National Science Foundation's I-Corps "train-the-trainer" program will be offered to up to 10 institutions supported through NCATS' Clinical and Translational Science Awards Program.

August 2015 — Supported by NCATS’ Tissue Chip for Drug Screening program, a team of scientists from Northwestern University, Charles Stark Draper Laboratory and the University of Illinois at Chicago (UIC) designed a miniaturized 3-D representation of the female reproductive tract and liver – called EVATAR™ – on a handheld, interconnected platform for use in drug testing and to study the basic biology of female reproduction.

September 2015 — NCATS announced it would spearhead the second phase of several NIH ExRNA Communication program projects to test and validate exRNA molecules for their potential as disease biomarkers and treatments. The ExRNA Communications program is supported through the NIH Common Fund.

October 2015 — Anna L. Ramsey-Ewing, Ph.D., joined NCATS as director of the Office of Grants Management and Scientific Review.

October 2015 — NCATS announced that it is accepting proposals on a rolling basis to collaborate with Bridging Interventional Development Gaps (BrIDGs) and Therapeutics for Rare and Neglected Diseases (TRND) program scientists.

NCATS Legislative Chronology

December 23, 2011 — President Obama signed into law the Consolidated Appropriations Act, 2012 (P.L. 112-74), enabling NIH to establish NCATS. This law also transferred authority over the Cures Acceleration Network (CAN) to NCATS. Authorized to reduce significant barriers to successful translation and accelerate the development of high-need cures, the CAN provides NCATS with flexibility in how it funds projects. Implementation of this authority is guided by the CAN Review Board.

Biographical Sketch of NCATS Director Christopher P. Austin, M.D.

On September 14, 2012, NIH Director Francis S. Collins announced the appointment of Christopher P. Austin, M.D., as director of the National Center for Advancing Translational Sciences. Austin succeeded former acting director of NCATS and current director of the National Institute of Mental Health Thomas R. Insel, M.D., on September 23, 2012.Bill Branson

Christopher P. Austin, M.D., is director of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). Austin leads the Center’s work to improve the translation of observations in the laboratory, clinic and community into interventions that reach and benefit patients — from diagnostics and therapeutics to medical procedures and behavioral changes. Under his direction, NCATS researchers and collaborators are developing new technologies, resources and collaborative research models; demonstrating their usefulness; and disseminating the data, analysis and methodologies for use by the worldwide research community.

Austin’s career has spanned the spectrum of translational research in the public and private sectors. He joined NIH in 2002 as the senior advisor to the director for translational research at the National Human Genome Research Institute (NHGRI), where he was responsible for conceptualizing and implementing research programs to derive scientific insights and therapeutic benefits from the results of the newly completed Human Genome Project. While at NHGRI, Austin founded and directed the NIH Chemical Genomics Center (now the NCATS Chemical Genomics Center), Therapeutics for Rare and Neglected Diseases program, Toxicology in the 21st Century initiative, and NIH Center for Translational Therapeutics. When NCATS launched in late 2011, Austin became the inaugural director of the Center’s Division of Pre-Clinical Innovation, and then was appointed as the NCATS director in 2012. Before joining NIH, Austin worked at the pharmaceutical company Merck, where he directed programs on genome-based discovery of novel targets and drugs, with a particular focus on treatments for schizophrenia and Alzheimer’s disease.

Austin is trained as a clinician and geneticist, and he is a member of the National Academy of Medicine, formerly the Institute of Medicine. He earned an M.D. from Harvard Medical School and an A.B. summa cum laude in biology from Princeton University. He completed a research fellowship in developmental neurogenetics at Harvard, studying genetic and environmental influences on stem cell fate determination. Austin also trained in internal medicine and neurology at the Massachusetts General Hospital in Boston, after which he practiced medicine in academic and community hospitals, providing primary care in urban settings and in rural Alaska and Africa.

NCATS Directors

Name

In Office from

To

Thomas R. Insel (Acting)

December 23, 2011

September 22, 2012

Christopher P. Austin

September 23, 2012

Present

Major Programs

Assay Development and Screening Technology (ADST) program. ADST is designed to advance therapeutic development through research and development of innovative assay (test) designs and chemical library screening methods. Program experts work to optimize assays requested or submitted by the biomedical research community for high-throughput small-molecule screening.

Bridging Interventional Development Gaps (BrIDGs) program. BrIDGs supports research collaborations to advance candidate therapeutics for both common and rare diseases into clinical testing. Investigators selected through an application process partner with NCATS experts to generate pre-clinical data and clinical-grade material through government contracts for use in Investigational New Drug applications to a regulatory authority such as the Food and Drug Administration.

Chemistry Technology program. Chemistry technology experts at NCATS develop small molecules and screening approaches that other scientists can use to pursue innovations in therapeutic development. The aim is to provide cutting-edge resources for drug development that benefit all NCATS scientists and their many research partners.

Clinical and Translational Science Awards (CTSA) Program. The CTSA Program supports an innovative national network of medical research institutions to improve the clinical phases of translational research process, from the first time a new intervention is tested in humans to dissemination into medical practice in communities. CTSA Program research centers serve as hubs locally and regionally to catalyze innovation in clinical research and training. Program support enables researchers to share best practices and build an increasingly robust national capacity for rapid and effective translation of newly developed interventions into improved health outcomes.

Discovering New Therapeutic Uses for Existing Molecules (New Therapeutic Uses) program. Through this innovative program, NCATS aims to improve the process of developing new treatments and cures for disease by finding new uses for assets that already have cleared several key steps along the development path (also known as drug repurposing). Using a crowdsourcing approach, NCATS matches researchers with a selection of pharmaceutical industry assets to rapidly test new ideas for existing investigational compounds, with the ultimate goal of identifying promising new treatments for patients.

Extracellular RNA (exRNA) Communication program. Through this NIH Common Fund program, scientists are beginning to understand the potential exRNA research may hold for improving understanding, diagnosis, prognosis and treatment of a wide variety of diseases and conditions, such as cancer, bone marrow disorders, heart disease, Alzheimer’s disease and multiple sclerosis. ExRNA communication is a recently discovered cell-to-cell signaling process that holds enormous promise for improving our understanding of a wide variety of diseases.

Genetic and Rare Disease Information Center (GARD). This NCATS collaboration with the National Human Genome Research Institute offers comprehensive information on rare and genetic diseases to patients, their families, health care providers and the public. The online GARD database provides accurate, up-to-date information about ongoing research, symptoms, treatment options and other details.

Matrix Combination Screening. NCATS experts use a technology called matrix combination screening to quickly narrow down a long list of potential drug combinations and find those with the most potential to help patients. The matrix screening approach uses NCATS’ robotic, high-throughput screening platform to quickly conduct millions of tests to assess the effects of a combination of therapeutic compounds on cellular, molecular or biochemical processes that are relevant to a disease of interest.

NCATS Chemical Genomics Center (NCGC). NCGC researchers advance small molecule therapeutic development through assay (test) design, high-throughput screening and medicinal chemistry. Small molecule chemical compounds, which can be used to test or “probe” the effects of increasing or decreasing the activity of a biological target in cells or animals, are some of the most powerful tools for target validation, which is the process of demonstrating that engaging a target provides meaningful therapeutic benefit. Probes enable researchers to investigate protein, cell functions and biological processes. If appropriate, probes can be optimized to become potential drug candidates.

Pfizer’s Centers for Therapeutic Innovation (CTI) for NIH Researchers. This innovative collaboration is led by NCATS and designed to help bridge the gap between early scientific discovery and its translation into new medicines through public-private resource sharing. Pfizer’s CTI program pairs leading researchers with Pfizer resources to pursue scientific and medical advances through joint therapeutic development.

Rare Diseases Clinical Research Network (RDCRN). NCATS’ RDCRN program is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.

RNA interference (RNAi). NCATS’ RNAi program is designed to develop and improve RNAi screening approaches to better understand gene function and identify treatment targets. Gene silencing through RNAi has emerged as a powerful tool for understanding gene function. Over the past several years, high-throughput RNAi screens have illuminated a wide variety of biological processes, ranging from genes that affect the activity of therapeutic agents to novel components of signaling pathways.

Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR). These programs support NCATS’ mission to transform the translational science process by helping small businesses develop and commercialize new technologies so that new treatments and cures for disease can be delivered to patients more efficiently. They serve as an engine of innovation, offering grants, contracts and technical assistance to small businesses and research organizations focused on advancing translational research and technologies that will improve disease prevention, detection and treatment.

Stem Cell Translation Laboratory (SCTL). NCATS is working to advance translational methods in stem cell research with support from the NIH Common Fund. Induced pluripotent stem cells are particularly useful because scientists can transform them into many different cell types to use for research or therapies. Through the SCTL, NCATS will provide researchers across various disciplines and organizations with the ability to establish collaborations to advance the translation of regenerative medicine applications.

Therapeutics for Rare and Neglected Diseases (TRND) program. Through the TRND program, NCATS supports pre-clinical development of therapeutic candidates intended to treat rare or neglected disorders, with the goal of enabling an Investigational New Drug application. The mission of the program is to encourage and speed the development of new treatments for diseases with high unmet medical needs. TRND stimulates therapeutic development research collaborations among NIH and academic scientists, nonprofit organizations, and pharmaceutical and biotechnology companies working on rare and neglected illnesses.

Tissue Chip for Drug Screening (Tissue Chip) program. This NCATS collaboration with the Defense Advanced Research Projects Agency and FDA supports the development of bioengineered devices to improve the process of predicting whether drugs will be safe or toxic in humans. The focus is on 3-D platforms engineered to support living human tissues and cells, called tissue chips or organs-on-chips. Tissue chip devices are designed as accurate models of the structure and function of human organs, such as the lung, liver and heart. Once developed and integrated, researchers can use these models to predict whether a candidate drug, vaccine or biologic agent is safe or toxic in humans in a faster and more cost-effective way than current methods.

Toxicology in the 21st Century (Tox21) program. Tox21 is a federal collaborative effort among NIH — including NCATS and the National Toxicology Program at the National Institute of Environmental Health Sciences — the Environmental Protection Agency and the FDA. Tox21 researchers aim to develop better toxicity assessment methods to quickly and efficiently test whether certain chemical compounds have the potential to disrupt processes in the human body that may lead to negative health effects. Through Tox21, researchers are testing 10,000 drugs and environmental chemicals for their potential to affect molecules and cells in ways that can cause health problems. The compounds undergo testing in NCATS’ high-speed robotic screening system.